Promethera is looking to advance selected development candidates from its portfolio together with industry partners to increase the likelihood of success in development. These activities include the use of genetically modified liver-derived stem cells in hemophilia and other diseases, where Promethera’s cells can be used as a long-term, endogenous delivery system for the partner’s therapeutic candidate.
Additionally, Promethera is looking to secure regional or global commercial partnerships, as applicable around the Heparesc product line to maximize commercial success. Heparesc is currently under submission process in the indication Urea Cycle Disorder (UCD) at Health Canada.
Heparesc Partnering Strategy
The Heparesc product line, a liver cell suspension consisting of human liver-derived mature hepatocytes, was acquired as part of the Cytonet acquisition in 2016. Heparesc has been developed by Cytonet for the treatment urea cycle disorders (UCDs) in pediatric patients through the different stages of clinical development and is now at a pre-commercial stage.
Following the integration of Cytonet, Promethera will prioritize commercial partnerships around Heparesc. A first example of such an alliance was established in 2017, when Promethera signed a regional license and collaboration agreement with LifeLiver, the Korean leader in the field of treatment of severe liver diseases.
Gene-Transfer Augmented Therapeutic Cell Lines
Promethera has established a combo cell-gene therapy platform that can give rise to promising product candidates in hemophilia A and B and potentially other indications. These approaches use the clinically validated HepaStem product line.
Hemophilia is a genetic disorder that impairs the body's ability to make blood clots. Hemophilia patients bleed longer after an injury, bruise easily, and are an increased risk of internal bleeding.
Hemophilia remains a disease in search for a cure. While gene therapy has provided new hope for hemophilia patients, issues surrounding the durability of expression and immune responses against gene transfer vectors remain.
Promethera is pursuing a potential solution to this problem using HepaStem cells which are genetically engineered ex vivo with Factor VIII or Factor IX and then applied via intravenous infusion.
HepaStem consists of liver-derived mesenchymal stem cells (MSCs progenitor cells) isolated from normal adult human liver tissue. Safety has been shown in Phase I/II clinical trial in 20 young patients. Promethera has successfully generated HepaStem cells which highly express Factor IX. Following intravenous infusion in a hemophilia patient, homing of these cells to the liver as well as to a site with hemophilia-related arthropathy has been observed.
This cell-based gene therapy approach is currently in pre-clinical stage and is expected to allow physiological FVIII/FIX production as well as cell re-administration because of the low immunogenicity of MSCs.
For more information, please contact our Business Development team.